EP2475376A4 - FORMULATIONS FOR LYSOSOMAL ENZYMES - Google Patents

FORMULATIONS FOR LYSOSOMAL ENZYMES

Info

Publication number
EP2475376A4
EP2475376A4 EP10790224.9A EP10790224A EP2475376A4 EP 2475376 A4 EP2475376 A4 EP 2475376A4 EP 10790224 A EP10790224 A EP 10790224A EP 2475376 A4 EP2475376 A4 EP 2475376A4
Authority
EP
European Patent Office
Prior art keywords
formulations
lysosomal enzymes
lysosomal
enzymes
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
EP10790224.9A
Other languages
German (de)
French (fr)
Other versions
EP2475376A2 (en
EP2475376B1 (en
Inventor
Jonathan H Lebowitz
Byeong Chang
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biomarin Pharmaceutical Inc
Original Assignee
Biomarin Pharmaceutical Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Biomarin Pharmaceutical Inc filed Critical Biomarin Pharmaceutical Inc
Priority to EP19203438.7A priority Critical patent/EP3679942A1/en
Priority to EP16153694.1A priority patent/EP3075386B1/en
Publication of EP2475376A2 publication Critical patent/EP2475376A2/en
Publication of EP2475376A4 publication Critical patent/EP2475376A4/en
Application granted granted Critical
Publication of EP2475376B1 publication Critical patent/EP2475376B1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22Hormones
    • A61K38/30Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Endocrinology (AREA)
  • Dermatology (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
EP10790224.9A 2009-06-17 2010-06-17 Formulations for lysosomal enzymes Active EP2475376B1 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP19203438.7A EP3679942A1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP16153694.1A EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US18774709P 2009-06-17 2009-06-17
PCT/US2010/039083 WO2010148253A2 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Related Child Applications (3)

Application Number Title Priority Date Filing Date
EP16153694.1A Division EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP16153694.1A Division-Into EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP19203438.7A Division EP3679942A1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Publications (3)

Publication Number Publication Date
EP2475376A2 EP2475376A2 (en) 2012-07-18
EP2475376A4 true EP2475376A4 (en) 2013-05-22
EP2475376B1 EP2475376B1 (en) 2016-03-30

Family

ID=43357058

Family Applications (3)

Application Number Title Priority Date Filing Date
EP10790224.9A Active EP2475376B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP16153694.1A Active EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP19203438.7A Pending EP3679942A1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Family Applications After (2)

Application Number Title Priority Date Filing Date
EP16153694.1A Active EP3075386B1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes
EP19203438.7A Pending EP3679942A1 (en) 2009-06-17 2010-06-17 Formulations for lysosomal enzymes

Country Status (4)

Country Link
US (1) US8785168B2 (en)
EP (3) EP2475376B1 (en)
ES (2) ES2569514T3 (en)
WO (1) WO2010148253A2 (en)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2932982B1 (en) 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
HRP20170698T1 (en) * 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lysosomal directed peptides and their uses
AU2009262670B2 (en) 2008-06-26 2013-06-20 Zevra Denmark A/S Use of Hsp70 as a regulator of enzymatic activity
CA2817773A1 (en) 2010-11-30 2012-06-07 Orphazyme Aps Methods for increasing intracellular activity of hsp70
US20140377244A1 (en) * 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR20200032244A (en) * 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 High concentration alpha-glucosidase compositions for the treatment of pompe disease
HUE039334T2 (en) 2012-11-27 2018-12-28 Biomarin Pharm Inc Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
EP2981551B1 (en) 2013-02-20 2020-06-03 Valerion Therapeutics, LLC Methods and compositions for treatment of pompe disease
CN105431527A (en) * 2013-06-11 2016-03-23 比奥卡尔齐斯股份有限公司 Biomolecule drying process for long-term storage
US20160346363A1 (en) * 2013-07-15 2016-12-01 New York University Methods and oral formulations for enzyme replacement therapy of human lysosomal and metabolic diseases
IL312865B2 (en) 2013-09-11 2025-06-01 Eagle Biologics Inc Liquid protein formulations containing viscosity-reducing agents
KR102058864B1 (en) * 2013-12-16 2019-12-24 주식회사 티움바이오 Pharmaceutical composition having improved stability comprising a fusion protein of factor vii
AU2015317447B2 (en) 2014-09-15 2021-02-25 Zevra Denmark A/S Arimoclomol formulation
WO2016054231A1 (en) 2014-09-30 2016-04-07 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
EP3200804A4 (en) 2014-10-01 2018-04-18 Eagle Biologics, Inc. Polysaccharide and nucleic acid formulations containing viscosity-lowering agents
WO2016065319A1 (en) * 2014-10-24 2016-04-28 Shire Human Genetic Therapies, Inc. Lysosomal targeting of enzymes, and uses thereof
MA41022A (en) * 2014-11-24 2017-10-03 Shire Human Genetic Therapies LYSOSOMIAL TARGETING AND CORRESPONDING USES
US9981021B1 (en) 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof
KR20170134748A (en) 2015-04-14 2017-12-06 추가이 세이야쿠 가부시키가이샤 A medicament composition for preventing and / or treating atopic dermatitis containing IL-31 antagonist as an active ingredient
US11130815B2 (en) 2015-06-24 2021-09-28 Jcr Pharmaceuticals Co., Ltd. Fusion proteins containing a BDNF and an anti-human transferrin receptor antibody
KR102510941B1 (en) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 Enriched acid alpha-glucosidase for the treatment of Pompe disease
EP4275751A3 (en) * 2016-02-24 2023-12-27 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
CN109475607B (en) * 2016-03-30 2022-04-26 阿米库斯治疗学公司 Formulations comprising recombinant acid alpha-glucosidase
IL325155A (en) * 2016-03-30 2026-02-01 Amicus Therapeutics Inc Formulations comprising recombinant acid alpha-glucosidase
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
NZ748599A (en) 2016-04-21 2022-08-26 Baylor College Medicine Compositions and methods for the treatment of lysosomal storage disorders and disorders characterized by lysosomal dysfunction
DK3782624T3 (en) 2016-04-29 2025-12-15 Zevra Denmark As Arimoclomol for the treatment of glucocerebrosidase-associated disorders
AU2017385274B2 (en) 2016-12-26 2024-02-22 Jcr Pharmaceuticals Co., Ltd. Fusion protein including BDNF
TWI761413B (en) 2016-12-26 2022-04-21 日商Jcr製藥股份有限公司 Novel anti-human transferrin receptor antibodies that pass through the blood-brain barrier
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
FI3635009T3 (en) 2017-06-07 2026-04-07 Regeneron Pharma Compositions and methods for internalizing enzymes
JP7779653B2 (en) 2018-02-07 2025-12-03 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Methods and compositions for therapeutic protein delivery
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CN114127275A (en) * 2019-04-30 2022-03-01 宾夕法尼亚州大学信托人 Composition for treating pompe disease
PT4034115T (en) * 2019-09-25 2025-03-25 Lumosa Therapeutics Co Ltd Pharmaceutical composition comprising thrombolytic peptide-tetrahydroisoquinoline conjugate
CN114728064B (en) 2019-11-20 2025-05-06 中外制药株式会社 Antibody-containing preparations
WO2022104261A1 (en) * 2020-11-16 2022-05-19 Avrobio, Inc. Compositions and methods for treating pompe disease
KR20230128462A (en) 2020-11-19 2023-09-05 제브라 덴마크 에이/에스 Process for preparing arimoclomol citrate and intermediates thereof
US20240318156A1 (en) * 2021-06-25 2024-09-26 Biomarin Pharmaceutical Inc. Compositions of beta-hexosaminidase variants and uses thereof
EP4619443A2 (en) 2022-11-18 2025-09-24 Seismic Therapeutic, Inc. Fc fusion molecules and uses thereof
JP2026501731A (en) 2023-01-06 2026-01-16 サイズミック セラピューティク インコーポレイテッド Protease variants and uses thereof
CN120118891B (en) * 2025-03-10 2025-11-21 广州青囊生物科技有限公司 Beta-glucosidase freeze-drying protective agent and application thereof in production of CK

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040161837A1 (en) * 1995-08-02 2004-08-19 Reuser Arnold J. Methods of purifying human acid alpha-glucosidase
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20080014188A1 (en) * 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof

Family Cites Families (83)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4309776A (en) 1980-05-13 1982-01-12 Ramon Berguer Intravascular implantation device and method of using the same
US4749570A (en) 1981-12-31 1988-06-07 The Governors Of The University Of Alberta Targeting conjugates of albumin and therapeutic agents
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
JPS61502657A (en) 1984-07-13 1986-11-20 チロン コ−ポレイシヨン Preproinsulin-like growth factors 1 and 2
DE3679343D1 (en) 1985-03-28 1991-06-27 Chiron Corp EXPRESSION BY USING FUSION GENES FOR PROTEIN PRODUCTION.
US4801575A (en) 1986-07-30 1989-01-31 The Regents Of The University Of California Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US4902505A (en) 1986-07-30 1990-02-20 Alkermes Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US5470828A (en) 1987-12-24 1995-11-28 Gropep Pty. Ltd. Peptide analogs of insulin-like growth factor II
US5817623A (en) 1995-03-06 1998-10-06 Univ Colorado State Res Found Method for treating diabetic peripheral neuropathy with IGF-I or IGF-II
US5549892A (en) 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US6451600B1 (en) 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5977307A (en) 1989-09-07 1999-11-02 Alkermes, Inc. Transferrin receptor specific ligand-neuropharmaceutical agent fusion proteins
US6610299B1 (en) 1989-10-19 2003-08-26 Aventis Pharma Deutschland Gmbh Glycosyl-etoposide prodrugs, a process for preparation thereof and the use thereof in combination with functionalized tumor-specific enzyme conjugates
US5248606A (en) 1990-06-11 1993-09-28 Dowelanco Dna encoding inactive precursor and active forms of maize ribosome inactivating protein
US5401650A (en) 1990-10-24 1995-03-28 The Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of biologically active α-galactosidase A
US5356804A (en) 1990-10-24 1994-10-18 Mount Sinai School Of Medicine Of The City Of New York Cloning and expression of biologically active human α-galactosidase A
US5633235A (en) 1991-04-19 1997-05-27 Regents Of The University Of Michigan Triciribine and analogs as antiviral drugs
FR2676058B1 (en) 1991-04-30 1994-02-25 Hoechst Lab GLYCOSYLATED PRODUCTS, THEIR PREPARATION PROCESS AND THEIR USE IN THE TREATMENT OF CANCERS.
US5736363A (en) 1991-07-29 1998-04-07 British Bio-Technology Limited IGF-II analogues
US6270989B1 (en) 1991-11-05 2001-08-07 Transkaryotic Therapies, Inc. Protein production and delivery
US5258453A (en) 1992-01-21 1993-11-02 University Of Utah Drug delivery system for the simultaneous delivery of drugs activatable by enzymes and light
EP0646178A1 (en) 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
US5981194A (en) 1992-07-10 1999-11-09 University Of British Columbia Use of p97 and iron binding proteins as diagnostic and therapeutic agents
US5476779A (en) 1992-09-30 1995-12-19 University Of Maryland At College Park DNA encoding insulin-like growth factor II isolated from rainbow trout
EP0599303A3 (en) 1992-11-27 1998-07-29 Takeda Chemical Industries, Ltd. Peptide conjugate
US5633234A (en) 1993-01-22 1997-05-27 The Johns Hopkins University Lysosomal targeting of immunogens
PT682524E (en) * 1993-02-02 2002-03-28 Xoma Technology Ltd PHARMACEUTICAL COMPOSITIONS CONTAINING PERMEABILITY INDUCTIVE BACTERICID PROTEIN AND A TENSIOACTIVE AGENT
US5798366A (en) 1993-05-13 1998-08-25 Monsanto Company Method for treatment of CNS-involved lysosomal storage diseases
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5704910A (en) 1995-06-05 1998-01-06 Nephros Therapeutics, Inc. Implantable device and use therefor
US5817789A (en) 1995-06-06 1998-10-06 Transkaryotic Therapies, Inc. Chimeric proteins for use in transport of a selected substance into cells
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
US20020013953A1 (en) 1995-08-02 2002-01-31 Reuser Arnold J. Compositions and methods for treating enzyme deficiency
JP3713276B2 (en) 1995-09-29 2005-11-09 バイオマリン ファーマシューティカル インコーポレイテッド Use of heparinase to reduce inflammatory response
US6348194B1 (en) 1995-11-13 2002-02-19 Ixsys Incorporated Tumor specific internalizing antigens and methods for targeting therapeutic agents
US6344436B1 (en) 1996-01-08 2002-02-05 Baylor College Of Medicine Lipophilic peptides for macromolecule delivery
JP2000501744A (en) 1996-01-22 2000-02-15 クリエイティブ バイオモレキゥルズ,インコーポレーテッド Morphogen analogs and their preparation
US6458574B1 (en) 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US6020144A (en) 1996-09-12 2000-02-01 Symbiontics, Inc. Sustained delivery device comprising a Leishmania protozoa and methods of making and using the same
US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US6235874B1 (en) 1997-01-10 2001-05-22 Academia Sinica Production of biologically active recombinant insulin-like growth factor II polypeptides
US6329501B1 (en) 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
ATE359561T1 (en) 1997-06-02 2007-05-15 Univ Johns Hopkins COMPUTER METHODS USING FREE ENERGY CALCULATION FOR LIGAND DESIGN AND PREDICTION OF BINDING TARGETS
US6472140B1 (en) 1997-09-05 2002-10-29 The General Hospital Corporation α-2- macroglobulin therapies and drug screening methods for Alzheimer's disease.
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
WO2000009725A2 (en) 1998-08-11 2000-02-24 Biosource Technologies, Inc. Method for recovering proteins from the interstitial fluid of plant tissues
DK1137762T3 (en) 1998-12-07 2009-02-02 Genzyme Corp Treatment of Pompe disease
US6596500B1 (en) 1998-12-08 2003-07-22 The General Hospital Corporation Binding of retinoids to M6P/IGF-II receptor
US6638727B1 (en) 1999-01-26 2003-10-28 Cytyc Health Corporation Methods for identifying treating or monitoring asymptomatic patients for risk reduction or therapeutic treatment of breast cancer
US6770468B1 (en) 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6585971B1 (en) 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
US6569661B1 (en) 1999-11-12 2003-05-27 Biomarin Pharmaceutical Inc. Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US20020081654A1 (en) 2000-04-07 2002-06-27 Sandrin Mauro Sergio Targeting hydrolase enzymes
AU7194101A (en) 2000-07-18 2002-01-30 Univ Duke Treatment of glycogen storage disease type ii
US20020142299A1 (en) 2001-01-09 2002-10-03 Davidson Beverly L. PTD-modified proteins
US7723296B2 (en) 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
US20030004236A1 (en) 2001-04-20 2003-01-02 Meade Thomas J. Magnetic resonance imaging agents for detection and delivery of therapeutic agents and detection of physiological substances
ATE384736T1 (en) 2001-04-30 2008-02-15 Zystor Therapeutics Inc SUB-CELLULAR TARGETING OF THERAPEUTIC PROTEINS
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
WO2003032913A2 (en) 2001-10-16 2003-04-24 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US6800472B2 (en) 2001-12-21 2004-10-05 Genzyme Glycobiology Research Institute, Inc. Expression of lysosomal hydrolase in cells expressing pro-N-acetylglucosamine-1-phosphodiester α-N-acetyl glucosimanidase
US6905856B2 (en) 2001-12-21 2005-06-14 Genzyme Glycobiology Research Institute, Inc. Soluble GlcNAc phosphotransferase
AU2003224880A1 (en) 2002-04-05 2003-10-27 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
US7485314B2 (en) 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
CA2487815A1 (en) 2002-05-29 2003-12-11 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins
US20050142141A1 (en) 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
ATE521701T1 (en) 2003-01-22 2011-09-15 Univ Duke IMPROVED CONSTRUCTS FOR EXPRESSING LYSOSOMAL POLYPEPTIDES
US20050026823A1 (en) 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
CN1922313B (en) 2004-02-10 2011-10-26 生物马林医药公司 Acid alpha-glucosidase and fragments thereof
WO2005077333A2 (en) 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
US8017151B2 (en) 2004-09-07 2011-09-13 Board Of Regents Of The University Of Nebraska By And Behalf Of The University Of Nebraska Medical Center Amphiphilic polymer-protein conjugates and methods of use thereof
EP1888098A2 (en) 2005-05-25 2008-02-20 Neose Technologies, Inc. Glycopegylated erythropoietin formulations
US7713709B2 (en) 2006-03-17 2010-05-11 Biomarin Pharmaceutical Inc. Assay for detection of antibodies to lysosomal enzymes
WO2008109677A2 (en) 2007-03-06 2008-09-12 Saint Louis University Modified enzyme and treatment method
HRP20170698T1 (en) * 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lysosomal directed peptides and their uses

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040161837A1 (en) * 1995-08-02 2004-08-19 Reuser Arnold J. Methods of purifying human acid alpha-glucosidase
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20080014188A1 (en) * 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
PASTORES G M ET AL: "Enzyme-replacement therapy for Pompe disease", PEDIATRIC HEALTH, FUTURE MEDICINE LTD, vol. 3, no. 1, 1 January 2009 (2009-01-01), pages 41 - 49, XP009167802, ISSN: 1745-5111 *
See also references of WO2010148253A2 *

Also Published As

Publication number Publication date
US8785168B2 (en) 2014-07-22
WO2010148253A3 (en) 2011-05-26
EP3075386B1 (en) 2019-10-16
ES2569514T3 (en) 2016-05-11
WO2010148253A2 (en) 2010-12-23
EP2475376A2 (en) 2012-07-18
ES2758827T3 (en) 2020-05-06
US20120148556A1 (en) 2012-06-14
EP2475376B1 (en) 2016-03-30
EP3679942A1 (en) 2020-07-15
EP3075386A1 (en) 2016-10-05

Similar Documents

Publication Publication Date Title
EP2475376A4 (en) FORMULATIONS FOR LYSOSOMAL ENZYMES
HRP20181347T1 (en) TWO-TABLET FORMULATIONS
PT2451486T (en) PEGUILED L-ASPARAGINASE
EP2439812A4 (en) DIRECTIONAL COUPLER
EP2401367A4 (en) TRANSPOSASES PIGGYBAC HYPERACTIVES
SMT201500053B (en) 2-Aminobenzimidazol-5-carboxamides as native-inflammatory agents
ZA201106050B (en) Dye-polymers formulations
BRPI1016228A2 (en) Compatible electrolyte formulations
HUE039556T2 (en) Digestive enzyme preparation for eclampsia
EP2475761A4 (en) photobioreactor
DK2269920T3 (en) container coupling
DK3412771T3 (en) AMYLASE POLYPEPTIDER
EP2504020A4 (en) DAPTOMYCIN FORMULATIONS
FR2947376B1 (en) DISCONNECT.
ATE510641T1 (en) POURING NOZZLE
EP2519755A4 (en) COUPLING FACES
EP2478768A4 (en) ANTIPHYTOVIRAL AGENT
EP2516615A4 (en) IMPROVED BIOREACTORS
EP2483480A4 (en) COUPLER
GB0919210D0 (en) Formulations
BRPI1009018A2 (en) photobioreactor
BRPI1005162A2 (en) beverage
SMT202100150T1 (en) IMPROVED FORMULATIONS
EP2400840A4 (en) Liquid statin formulations
FR2952847B3 (en) >

Legal Events

Date Code Title Description
PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

17P Request for examination filed

Effective date: 20120426

AK Designated contracting states

Kind code of ref document: A2

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO SE SI SK SM TR

DAX Request for extension of the european patent (deleted)
A4 Supplementary search report drawn up and despatched

Effective date: 20130422

RIC1 Information provided on ipc code assigned before grant

Ipc: A61K 47/30 20060101ALI20130416BHEP

Ipc: A61P 3/00 20060101ALI20130416BHEP

Ipc: A61K 38/17 20060101ALI20130416BHEP

Ipc: A61K 47/34 20060101ALI20130416BHEP

Ipc: A61K 38/16 20060101AFI20130416BHEP

Ipc: A61K 38/46 20060101ALI20130416BHEP

17Q First examination report despatched

Effective date: 20140527

REG Reference to a national code

Ref country code: DE

Ref legal event code: R079

Ref document number: 602010031842

Country of ref document: DE

Free format text: PREVIOUS MAIN CLASS: A61K0038160000

Ipc: A61P0021000000

GRAP Despatch of communication of intention to grant a patent

Free format text: ORIGINAL CODE: EPIDOSNIGR1

INTG Intention to grant announced

Effective date: 20151002

RIC1 Information provided on ipc code assigned before grant

Ipc: A61P 3/00 20060101ALI20150924BHEP

Ipc: A61K 47/34 20060101ALI20150924BHEP

Ipc: A61P 21/00 20060101AFI20150924BHEP

Ipc: A61K 47/30 20060101ALI20150924BHEP

Ipc: A61K 38/46 20060101ALI20150924BHEP

Ipc: A61K 38/17 20060101ALI20150924BHEP

Ipc: A61K 38/16 20060101ALI20150924BHEP

GRAS Grant fee paid

Free format text: ORIGINAL CODE: EPIDOSNIGR3

GRAA (expected) grant

Free format text: ORIGINAL CODE: 0009210

AK Designated contracting states

Kind code of ref document: B1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO SE SI SK SM TR

REG Reference to a national code

Ref country code: GB

Ref legal event code: FG4D

REG Reference to a national code

Ref country code: CH

Ref legal event code: EP

REG Reference to a national code

Ref country code: AT

Ref legal event code: REF

Ref document number: 784716

Country of ref document: AT

Kind code of ref document: T

Effective date: 20160415

REG Reference to a national code

Ref country code: IE

Ref legal event code: FG4D

REG Reference to a national code

Ref country code: CH

Ref legal event code: NV

Representative=s name: FIAMMENGHI-FIAMMENGHI, CH

REG Reference to a national code

Ref country code: ES

Ref legal event code: FG2A

Ref document number: 2569514

Country of ref document: ES

Kind code of ref document: T3

Effective date: 20160511

REG Reference to a national code

Ref country code: DE

Ref legal event code: R096

Ref document number: 602010031842

Country of ref document: DE

REG Reference to a national code

Ref country code: FR

Ref legal event code: PLFP

Year of fee payment: 7

REG Reference to a national code

Ref country code: NL

Ref legal event code: FP

REG Reference to a national code

Ref country code: LT

Ref legal event code: MG4D

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: GR

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160701

Ref country code: HR

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: NO

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160630

Ref country code: FI

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

REG Reference to a national code

Ref country code: AT

Ref legal event code: MK05

Ref document number: 784716

Country of ref document: AT

Kind code of ref document: T

Effective date: 20160330

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: SE

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: LV

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: LT

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: EE

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: PL

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: IS

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160730

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: CZ

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: SK

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: PT

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160801

Ref country code: SM

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: AT

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: RO

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

REG Reference to a national code

Ref country code: DE

Ref legal event code: R097

Ref document number: 602010031842

Country of ref document: DE

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: DK

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: MC

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

PLBE No opposition filed within time limit

Free format text: ORIGINAL CODE: 0009261

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: NO OPPOSITION FILED WITHIN TIME LIMIT

26N No opposition filed

Effective date: 20170103

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: SI

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

REG Reference to a national code

Ref country code: FR

Ref legal event code: PLFP

Year of fee payment: 8

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: HU

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT; INVALID AB INITIO

Effective date: 20100617

Ref country code: CY

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

REG Reference to a national code

Ref country code: FR

Ref legal event code: PLFP

Year of fee payment: 9

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: MK

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

Ref country code: MT

Free format text: LAPSE BECAUSE OF NON-PAYMENT OF DUE FEES

Effective date: 20160630

Ref country code: LU

Free format text: LAPSE BECAUSE OF NON-PAYMENT OF DUE FEES

Effective date: 20160617

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: BG

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

PG25 Lapsed in a contracting state [announced via postgrant information from national office to epo]

Ref country code: AL

Free format text: LAPSE BECAUSE OF FAILURE TO SUBMIT A TRANSLATION OF THE DESCRIPTION OR TO PAY THE FEE WITHIN THE PRESCRIBED TIME-LIMIT

Effective date: 20160330

PGFP Annual fee paid to national office [announced via postgrant information from national office to epo]

Ref country code: NL

Payment date: 20250520

Year of fee payment: 16

PGFP Annual fee paid to national office [announced via postgrant information from national office to epo]

Ref country code: DE

Payment date: 20250520

Year of fee payment: 16

PGFP Annual fee paid to national office [announced via postgrant information from national office to epo]

Ref country code: GB

Payment date: 20250520

Year of fee payment: 16

PGFP Annual fee paid to national office [announced via postgrant information from national office to epo]

Ref country code: BE

Payment date: 20250520

Year of fee payment: 16

Ref country code: IT

Payment date: 20250520

Year of fee payment: 16

PGFP Annual fee paid to national office [announced via postgrant information from national office to epo]

Ref country code: FR

Payment date: 20250520

Year of fee payment: 16

PGFP Annual fee paid to national office [announced via postgrant information from national office to epo]

Ref country code: TR

Payment date: 20250526

Year of fee payment: 16

PGFP Annual fee paid to national office [announced via postgrant information from national office to epo]

Ref country code: IE

Payment date: 20250522

Year of fee payment: 16

PGFP Annual fee paid to national office [announced via postgrant information from national office to epo]

Ref country code: ES

Payment date: 20250701

Year of fee payment: 16

PGFP Annual fee paid to national office [announced via postgrant information from national office to epo]

Ref country code: CH

Payment date: 20250701

Year of fee payment: 16